BioMarin seeks approval to market another drug for rare disease

San Rafael-based BioMarin Pharmaceutical Inc., which develops medications for treatment of rare diseases, announced Monday it is seeking approval to begin marketing its latest drug in the United States.

The company estimates that approximately 3,000 people worldwide may suffer from the genetic disorder that its new drug candidate, Vimizim, will treat.

BioMarin, and the rapidly growing number of companies that sell drugs to treat such rare afflictions, known as "orphan diseases," are able to prosper financially despite marketing their products to a relatively small number of people because they are able to charge a lot for their medications.

In the case of Vimizim, "we haven't yet set a price," said Jeff Ajer, BioMarin's chief commercial officer. "But it is likely to be between $250,000 to $400,000 per year per patient."

In 2010, Forbes magazine ranked one of BioMarin's four approved products, Naglazyme, among the nine most expensive medications in the world. Forbes estimated Naglazyme cost $365,000 per year per patient.

Ajer said, "That's an OK estimate."

In 2012, net revenue from BioMarin's four approved products grew nearly 14 percent to about $497 million. The company, however, reported a net loss of $53 million due primarily, it said, to research and development expenses. It has six other products in development.

BioMarin has asked the U.S. Food and Drug Administration to issue it a biologics license for Vimizim, an enzyme replacement therapy for the treatment of Marquio A syndrome. The disease results in excessive storage of glycosaminoglycans — long chains of sugar carbohydrates — in cells that help build bone, cartilage, tendons, corneas, skin and connective tissue.

Ajer said one of the factors BioMarin considers when pricing its drugs is what kind of benefit they will deliver to patients. "We're generally looking at drugs that make a big impact on the lives of patients who have these disorders," he said.

The disorder that Naglazyme treats affects mostly children, about 1,200 worldwide, and can cause developmental delay, movement disorders, seizures, dementia, deafness and blindness. Ajer said the impact on drug companies and the overall cost of health care is diluted because so few people suffer from the disorders that BioMarin's drugs treat.

Since passage of the Orphan Drug Act in 1983, however, the number of companies producing orphan drugs has proliferated. The act gave orphan drug developers exclusive sales rights for seven years plus tax incentives for conducting clinical trials.

"Everybody is going after orphan drugs because they get approved really quickly, and you can make a lot of money even if you have only 10 patients," said Rhonda Greenapple, founder of Reimbursement Intelligence, a pharmaceutical market research firm in Madison, N.J. that surveys insurers.

Greenapple said it's illegal for U.S. insurance companies to refuse to pay for the type of ultra-orphan drug treatments that BioMarin produces. She predicts that eventually, however, insurance companies will try to cut deals with pharmaceutical companies to lower prices or shift part of the cost to patients.

"The question is when is enough, enough," Greenapple said. "It's getting out of control right now."